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Directed Differentiation and Disease Modeling

Human pluripotent stem cell (hPSC)-based models hold tremendous potential for studying human development and disease. Directed differentiation and disease modeling are two key methods for using hPSCs in drug discovery, cell therapy validation, and disease research.

  • Directed differentiation of hPSCs refers to the in vitro differentiation of these cells toward a specific cell type through defined cell culture conditions. Directed differentiation is achieved by the addition of specific growth factors or small molecules.
  • Disease modeling is an approach to study diseases using cells that display relevant pathological features. Disease modeling using hPSCs can be achieved by reproduction of a disorder-associated mutation with gene editing, isolation of embryonic stem cells (ESCs) from affected blastocysts; or generation of induced pluripotent stem cells (iPSCs) from patients’ somatic cells.

Explore the resources below to support your disease modeling studies using hPSCs.

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  1. Advancing Neurological and Retinal Research with Genetically Diverse Human iPSC-Derived Models
    Advancing Neurological and Retinal Research with Genetically Diverse Human iPSC-Derived Models
    In this webinar, Andrew Gaffney, Director of Stem Cell Manufacturing and Commercialization at º£½ÇÆÆ½â°æ Technologies, showcases how high-quality, genetically diverse iPSC lines and differentiated cells are advancing research in neurological and retinal diseases. Manufactured to meet ISSCR standards, these iPSC lines undergo extensive quality control, ensuring consistency and reliability for disease modeling and drug discovery. º£½ÇÆÆ½â°æ has optimized the differentiation of these iPSCs into mature, functional astrocytes and retinal pigment epithelial (RPE) cells that exhibit hallmark physiological behaviors.<br><br>As a supporter of, and participant in, ISSCR’s 2025 Annual Meeting, we share with you our presentation at ISSCR 2025. This presentation does not represent an endorsement from or support of the ISSCR.
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